First Patient Dosed in the Phase 1 Trial of PulseSight Therapeutics’ Investigational PST-611 Treatment for Dry AMD/GA
PulseSight Therapeutics has announced that the first patient has been dosed in its phase 1 clinical trial (PST-611-CT1) assessing the safety and tolerability of its lead program, PST-611, in humans.
PST-611 is a first-in-class non-viral vectorized therapy for the treatment of dry age-related macular degeneration (AMD)/geographic atrophy (GA), expressing human transferrin, a highly potent iron regulator, playing a central role in restoring normal iron homeostasis.
PST-611-CT1 is a first-in-human single ascending dose study that aims to establish, in 6 to 12 dry AMD/GA patients, the safety profile of the drug and validate the maximal tolerated dose in view of the following phase 2a proof-of-concept trial. Preliminary results are anticipated early 2026, subject to patient recruitment.
The study is being conducted in Paris and Grenoble by Professor Francine Behar-Cohen, MD, PhD at the Department of Ophthalmology, Cochin – Assistance Publique-Hôpitaux de Paris (AP-HP), the inventor of our technology, and Professor Christophe Chiquet, MD, PhD at the Department of Ophthalmology, CHU Grenoble Alpes.
“Having pioneered the development of the electro-transfection technology that delivers DNA plasmids encoding therapeutic proteins into the ciliary muscle of the eye, I am very excited to move PST-611, expressing transferrin, into its first clinical trial," Professor Francine Behar-Cohen, said in a company news release. "Late-stage dry AMD/GA is a progressing disease that leads to vision loss and for which we have no therapeutic options for our patients. Based on its mechanism of action and thanks to the innovative delivery technology, PST-611 has potential to become a major treatment option for these patients.”
“The dosing of the first patient in our PST-611-CT1 trial is a very exciting milestone for the company," said Judith Greciet, CEO of PulseSight Therapeutics. "Supported by the previous clinical demonstration of the safety profile of our innovative delivery technology and a solid preclinical package, we believe PST-611 holds the potential to improve both anatomical and functional features of dry AMD/GA. Moreover, the sustained and long-lasting expression of transferrin should help reduce the need for frequent reinjections, strongly improving patients’ compliance to the treatment. Once the safety and the maximal dose are confirmed, our goal is to swiftly move into a phase 2a proof-of-concept study, to demonstrate the ability of transferrin to protect retinal cells from atrophy and preserve vision.”